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RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
专利权人:
James McSwiggen;Leonid Beigelman
发明人:
James McSwiggen,Leonid Beigelman
申请号:
US10720448
公开号:
US08273866B2
申请日:
2003.11.24
申请国别(地区):
US
年份:
2012
代理人:
摘要:
The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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